‘Massive urgency’ for drug for DMD says Join Our Boys
George and Isaac Naughton, students at Roscommon Community College, at the launch by the college of a fundraising drive for them last year.
A Roscommon family is determined to continue its campaign for access to the drug Givinostat for people with Duchenne muscular dystrophy (DMD).
Twin brothers George and Isacc Naughton, both of whom have the devastating condition and their mother Paula recently met the Minister for Health Jennifer O’Carroll MacNeill, but there was no update on making the drug available. Currently, the State recommends that the drug not be considered for reimbursement unless its cost effectiveness can be improved.
The brothers turned 16 years old at the start of the month, the same age as their older brother Archie was when he passed away from DMD in July 2022.
Speaking to the Herald, Paula said that the drug had been licensed by the European Medicines Agency (EMA) for children who are six or over, on cortical steroids, and who can still walk. However, in the US the drug can be used for all children and young men with Duchenne.
“The EMA took a very different approach and we were obviously very disappointed by that decision,” she said. “The Join Our Boys Trust is very anxious that Givinostat is reimbursed and made available to every child over the age of six that can still stand or walk. Even though this will not benefit George and Isaac, we desperately want other children to get the best chance to live.” Paula said that there are approximately 55 children in Ireland, eligible for this drug.
“There is a massive urgency for this,” she said. She added that data on the drug's effectiveness for children with DMD was available in the first week of July 2022.
“We were even able to tell Archie this just before he died. But four years later and still the drug isn’t available to the children of Ireland,” she said, and she called for the whole process of drug reimbursement to be changed.
Paula said that the minister was very invested in trying to support the Duchenne community in this country.
“Givinostat was evaluated in just 22 days, the fastest an assessment has ever happened, and that was purely because of her intervention. The minister has to be commended for that but there is still so much work that needs to be done.”
